EP3384015A4 - Skalierbare verfahren zur herstellung eines rekombinanten adeno-assoziierten viralen vektors in einem für die klinische anwendung geeigneten serumfreien suspensionszellkultursystem - Google Patents
Skalierbare verfahren zur herstellung eines rekombinanten adeno-assoziierten viralen vektors in einem für die klinische anwendung geeigneten serumfreien suspensionszellkultursystem Download PDFInfo
- Publication number
- EP3384015A4 EP3384015A4 EP16871497.0A EP16871497A EP3384015A4 EP 3384015 A4 EP3384015 A4 EP 3384015A4 EP 16871497 A EP16871497 A EP 16871497A EP 3384015 A4 EP3384015 A4 EP 3384015A4
- Authority
- EP
- European Patent Office
- Prior art keywords
- aav
- serum
- vector
- cell culture
- system suitable
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/0018—Culture media for cell or tissue culture
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0603—Embryonic cells ; Embryoid bodies
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0684—Cells of the urinary tract or kidneys
- C12N5/0686—Kidney cells
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/70—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
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- C12N2510/00—Genetically modified cells
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Analytical Chemistry (AREA)
- Urology & Nephrology (AREA)
- Gynecology & Obstetrics (AREA)
- Developmental Biology & Embryology (AREA)
- Reproductive Health (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562261815P | 2015-12-01 | 2015-12-01 | |
| PCT/US2016/064414 WO2017096039A1 (en) | 2015-12-01 | 2016-12-01 | Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| EP3384015A1 EP3384015A1 (de) | 2018-10-10 |
| EP3384015A4 true EP3384015A4 (de) | 2019-05-29 |
Family
ID=58797705
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP16871497.0A Pending EP3384015A4 (de) | 2015-12-01 | 2016-12-01 | Skalierbare verfahren zur herstellung eines rekombinanten adeno-assoziierten viralen vektors in einem für die klinische anwendung geeigneten serumfreien suspensionszellkultursystem |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20190292561A1 (de) |
| EP (1) | EP3384015A4 (de) |
| JP (2) | JP7444521B2 (de) |
| KR (1) | KR20180091863A (de) |
| CN (1) | CN108603174A (de) |
| AU (3) | AU2016362317B2 (de) |
| CA (1) | CA3006309A1 (de) |
| CO (1) | CO2018006699A2 (de) |
| IL (1) | IL259595B2 (de) |
| MX (2) | MX2018006682A (de) |
| MY (1) | MY205245A (de) |
| PE (2) | PE20181534A1 (de) |
| PH (1) | PH12018501168B1 (de) |
| RU (1) | RU2766583C2 (de) |
| SG (2) | SG11201804400SA (de) |
| WO (1) | WO2017096039A1 (de) |
Families Citing this family (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3151866B1 (de) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimäre kapside |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| EP3384015A4 (de) * | 2015-12-01 | 2019-05-29 | Spark Therapeutics, Inc. | Skalierbare verfahren zur herstellung eines rekombinanten adeno-assoziierten viralen vektors in einem für die klinische anwendung geeigneten serumfreien suspensionszellkultursystem |
| EP3448874A4 (de) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | Zusammensetzungen zur behandlung einer erkrankung |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| EP3510161A4 (de) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | Zusammensetzungen und verfahren zur behandlung von nicht altersbedingten hörschäden bei einer person |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | ハンチントン病治療組成物および方法 |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| PE20200737A1 (es) * | 2017-06-30 | 2020-07-23 | Spark Therapeutics Inc | Metodos de purificacion de columna de vector aav |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| EP3808849A1 (de) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Zusammensetzungen und verfahren zur aav-freisetzung |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| EP3697908A1 (de) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Behandlung von amyotropher lateralsklerose (als) |
| MX2020003888A (es) * | 2017-10-18 | 2020-11-06 | Regenxbio Inc | Anticuerpos terapeuticos postraduccionalmente modificados completamente humanos. |
| JP2021505610A (ja) * | 2017-12-05 | 2021-02-18 | アプライド ジェネティック テクノロジーズ コーポレイション | ウイルス粒子のための製剤最適化 |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| EP3774852A1 (de) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antikörpermeidende virale vektoren |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| WO2019217483A1 (en) * | 2018-05-07 | 2019-11-14 | Spark Therapeutics, Inc. | Plasmid free aav vector producing cell lines |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| KR20210032984A (ko) * | 2018-07-11 | 2021-03-25 | 박스알타 인코퍼레이티드 | Aav 조성물 |
| CN108866011A (zh) * | 2018-07-31 | 2018-11-23 | 深圳生生凡非基因技术有限公司 | 一种同步包装rAAV的方法 |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| EP3856762A1 (de) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin-expressionskonstrukte mit gentechnisch veränderten promotoren und verfahren zu deren verwendung |
| CN110437317B (zh) * | 2019-01-30 | 2023-05-02 | 上海科技大学 | 具有变异衣壳蛋白的腺相关病毒及其用途 |
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| WO2021028837A1 (en) | 2019-08-13 | 2021-02-18 | Waters Technologies Corporation | Affinity resins and sample preparation devices based on cartilaginous fish ignar derived binding domains |
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| CN110894494B (zh) * | 2019-11-22 | 2022-09-27 | 广西梧州制药(集团)股份有限公司 | 一种大规模高密度悬浮培养293细胞高产腺病毒的方法 |
| CN110938654A (zh) * | 2019-12-11 | 2020-03-31 | 和元生物技术(上海)股份有限公司 | 一种细胞转染试剂及其应用 |
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| KR20220157944A (ko) | 2020-02-21 | 2022-11-29 | 아카우오스, 인크. | 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법 |
| KR20220166825A (ko) * | 2020-04-10 | 2022-12-19 | 사우스웨스트 리서치 인스티튜트 | 바이러스 벡터 생산을 위한 3차원 생물 반응기 |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| WO2022043926A1 (en) * | 2020-08-31 | 2022-03-03 | Intas Pharmaceuticals Ltd. | Process for preparation of recombinant adeno-associated virus particle |
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